Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.
Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment
Posted on: 23 December 2020, source: Science
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
Gene Therapy in One Eye Improves Vision in Both Eyes
Posted on: 12 December 2020, source: The Scientist
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye. In a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.
NIH project aims to maximize efficiency of AAV platform for gene therapy
Posted on: 19 November 2020, source: News-Medical.Net
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project is reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 16, 2020. "PaVe-GT is an experimental translational science initiative that aims to leverage the power of platform vectors and disease relatedness to help deliver on the promise of gene therapy to patients with rare diseases," states the NIH PaVe-GT team.
Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project is reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 16, 2020. "PaVe-GT is an experimental translational science initiative that aims to leverage the power of platform vectors and disease relatedness to help deliver on the promise of gene therapy to patients with rare diseases," states the NIH PaVe-GT team.
COVID-19, manufacturing challenges limit cell and gene therapy progress, FDA official says
Posted on: 30 October 2020, source: FDA/Healio.com
Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research. Peter Marks, MD, PhD, delivered this assessment during the virtual Cell & Gene Meeting on the Mesa.
Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research. Peter Marks, MD, PhD, delivered this assessment during the virtual Cell & Gene Meeting on the Mesa.
Gene Therapy in Mesothelioma Patients
Posted on: 28 September 2020, source: mesothelioma.com
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms.
Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms.
Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.
FDA grants orphan drug designation for LHON gene therapy
Posted on: 26 September 2020, source: FDA
Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.
Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.
New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients
Posted on: 8 September 2020, source: BioSpace
The National Institutes of Health (NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that.
The National Institutes of Health (NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that.
Gene Therapy News
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Development 'cookbooks' could speed up gene therapy - The Science Board
Jan 14, 2021 | 21:18 pm
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New gene therapy methods deliver promise - Spectrum
Jan 14, 2021 | 20:23 pm
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News briefing: Precision Medicine Group acquires cell and gene therapy-focused services firm Project Farma; RoosterBio and Sartorius collab on gene therapy manufacturing - Endpoints News
Jan 14, 2021 | 13:20 pm
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Center for cell and gene therapy to open next year - Harvard Gazette
Jan 14, 2021 | 07:00 am
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5 questions facing gene therapy in 2021 - BioPharma Dive
Jan 8, 2021 | 18:26 pm
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